Assessing a new treatment for Alpha-1 Antitrypsin Deficiency

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This open-label study evaluates the safety and tolerability of WVE-006 in participants with Alpha-1 Antitrypsin Deficiency (AATD) through both single and multiple ascending doses. The study aims to understand the pharmacodynamics and pharmacokinetics of the treatment in individuals with mild to moderate AATD-induced lung or liver disease. Participants will be monitored for their response to the treatment and any adverse effects that may arise during the dosing periods.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Healthy as determined by the Investigator, based on a medical evaluation, or has mild to moderate AATD-induced lung disease (FEV1 ≥50%) and/or stable mild AATD-induced liver disease (≤F2 (≤10 kPa) on FibroScan.
* Genetic testing confirming Pi\*ZZ.
* Participant has been a non-smoker for at least 1 year prior to screening.

Exclusion Criteria:

* • Participant has a history of multiple drug allergies or of allergic reaction to an oligonucleotide or to N-acetylgalactosamine (GalNAc).

  * Participant has a history of intolerance or any medical condition that might interfere with subcutaneous injections.
  * Any ongoing or recent infections.
  * Any recent or planned vaccinations during the study.
  * Participant has a history of regular alcohol consumption exceeding 14 standard drinks/week.
  * Unwilling to abstain from alcohol for 48 hours prior to dosing at each of the dosing visits.
  * Any recent or planned major surgery during the study.
  * Participant has any medical condition or social circumstance that, in the opinion of the Investigator, would make the participant unsuitable for participation in the study or for dosing on Day 1, or could interfere with the assessments of safety, pharmacodynamics, or pharmacokinetics, or completion of the study.
  * Participant currently on AAT augmentation therapy, planned to be on augmentation therapy anytime during the study, or has been on augmentation therapy within 30 days prior to Screening Visit.
  * Donation of blood or blood products in excess of 500 mL within 12 weeks prior to Screening Visit and/or unwilling to refrain from blood donation for the duration of the study.
  * Participant has received an investigational agent within 3 months of the Screening Visit.

Where it's running

• Inspiration Research Limited
  Toronto, Ontario, Canada Recruiting
• St. Vincent's Hospital, Melbourne
  Fitzroy, Victoria, Australia Active not recruiting
• Dalhousie University - Queen Elizabeth II Health Sciences Centre
  Halifax, Nova Scotia, Canada Active not recruiting
• Turku University Hospital
  Turku, Finland Active not recruiting
• Universitaetsklinikum Aachen, AoeR
  Aachen, Germany Active not recruiting
• Waikato Hospital
  Hamilton, New Zealand Active not recruiting

Study contacts

• Study coordinator: Clinical Operations
• Email: Clinicaltrials@wavelifesci.com
• Phone: 855-215-4687

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Prediction score for death or lung transplant in emphysema caused by alpha-1 antitrypsin deficiency

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• Two weekly doses of Alpha1-Proteinase Inhibitor by injection compared with standard IV dose in alpha-1 antitrypsin deficiency

  A 'how it works' test to see if two weekly subcutaneous doses are as safe and help the body process the medicine as well as the standard IV dose for adults with AAT deficiency.

  Recruiting at 10 sites Phase 3 Grifols Therapeutics LLC · Birmingham, Alabama and 21 other locations · NCT07555483

• AIR-001 treatment in adults with alpha-1 antitrypsin deficiency

  This Phase 1 trial will test single ascending and multiple doses of AIR-001 for safety and how the drug is processed in adults with PiZZ alpha-1 antitrypsin deficiency.

  Recruiting at 11 sites Phase 1 AIRNA Corporation · Melbourne and 12 other locations · NCT07431112

• Gene therapy using AAV8 to replace defective alpha-1 antitrypsin

  This trial will try a one-time IV gene therapy (AAV8hAAT(AVL)) to produce an oxidation-resistant AAT protein in adults with ZZ or Z/null alpha-1 antitrypsin deficiency who have emphysema.

  Recruiting Phase 1 Weill Medical College of Cornell University · New York, New York · NCT06996756

• PiMZ Longitudinal Cohort: tracking lung changes in PiMZ Alpha-1 patients

  This project will test whether CT lung density and blood or sputum biomarkers can predict worsening emphysema over three years in adults with PiMZ alpha-1 antitrypsin deficiency and COPD.

  Recruiting Columbia University · Birmingham, Alabama and 4 other locations · NCT06505603

• Evaluating BEAM-302 for treating Alpha-1 Antitrypsin Deficiency in adults

  This study is testing a new treatment called BEAM-302 to see if it can help adults with Alpha-1 Antitrypsin Deficiency who have lung or liver problems.

  Recruiting at 12 sites Phase 1/2 Beam Therapeutics Inc. · Birmingham, Alabama and 11 other locations · NCT06389877

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.